Background: Every patient with Parkinson’s disease deserves the highest quality care. This concept has inspired the National Parkinson Foundation’s efforts since its founding. Recent research in the United States has shown that neurologist care improves the odds of avoiding hip fracture, staying out of a nursing home, and avoiding death by approximately 20%. NPF has been focused on changing those odds through a detailed study of the care delivered in expert clinics through our observational clinical study, the Parkinson’s Outcomes Project. We have a new opportunity: because of advances in the management of healthcare, we can today set evidence-based standards for care delivery.

Clinical trials in which today’s new care is proven are always conducted with a protocols for care – not just the use of the drug which is being studied, but most other aspects of care. These protocols include instructions to standardize decisions about care, including when the patient’s status has changed enough that they should be taken off the protocol. Brent James, MD, PhD, started his career writing just such protocols for clinical trials, and then decided to take this experience into his routine care. Dr. James, working with Intermountain Health, created a protocol that recommended that doctors start with a basic model of care and modify it to suit their patients. Using this approach, James was able to achieve a reduction in early induction of labor by over 90% and ventilator use in premature babies by 75%. In 2014, Dr. James spoke at the NPF Centers Leadership Conference. He said to us, “You can do this.”

Methods/Design: In 2009, NPF launched the Parkinson’s Outcomes Project, a longitudinal outcomes registry tracking demographics, treatments, and outcomes in over 8,500 individuals treated at 21 academic medical centers. Similar to efforts that have revolutionized cystic fibrosis care and cardiovascular surgery, POP was designed to improve Parkinson’s care. It is now the largest study ever conducted in Parkinson’s disease. The main focus of this observational clinical trial is to link care delivered to long-term patient outcomes. The study has already been used to identify expert care practices for the management of depression and created a guideline for exercise. For this initiative, we have started the efforts to optimize the approach to the primary goal of medical management: dopamine replacement.

Over the last six months, the data set for the study has been expanded to include specific medications and doses. Data from the first seven years of the study was used to identify common medication patterns (presented at MDS in Berlin in June) and the association between medication patterns and better outcomes (presented at WPC in Portland, Oregon, in September). With the new dataset, the analysis team will examine how both medication choice and dosing impact outcomes. With eight years of outcomes data together with one year of data on specific use of medications and dosing, we will convene an expert panel of statisticians, machine learning experts, and leading movement disorders neurologists to distill the data into a candidate set of recommendations for how to select medications and set dosages for individuals with Parkinson’s to optimize response.

Relevance to Diagnosis/Treatment of Parkinson’s disease: We intend this project to establish standards of care that can be replicated across the world. Pairing evidence-based recommendations on basic Parkinson’s management with the resources to collect data will enable us to track outcomes internationally. By equipping academic medical centers with the methods utilized in the Parkinson’s Outcomes Project we can expand outcome tracking exponentially and thus improve our understanding of the best treatment protocols. Scientists at NPF and at academic medical centers affiliated with NPF would be able to review reports and deliver to our North American clinical partners the same model for continuous improvement of care that we have adopted in our own centers.

September 2017 Project Update:

The first analysis from this effort focused on identifying the highest-powered interventions, i.e., those interventions that, upon initiation, are linked to the most significant change in patient status. Utilizing a hypothesis-free “big data” analysis technique, the intervention associated with the largest improvement in patient status was identified to be Deep Brain Stimulation (DBS). Although DBS is not a medication, it is a treatment for Parkinson’s that includes numerous complexities: lead placement, surgical outcomes, programming, and re-balancing of medications in the new context. The Parkinson’s Outcomes Project steering committee decided that given the importance of DBS in patient outcomes, we couldn’t examine medications without considering DBS outcomes.

The Parkinson’s Outcomes Project included 499 participants who were recruited into the study prior to being recommended for DBS surgery and 10 centers had at least 25 participants who had DBS devices implanted (range: 28-48 participants), allowing us to compare outcomes across those sites. Among those ten centers, one center achieved, on average, an 8 point improvement in patient’s health related quality of life scores (HRQL), measured with the PDQ-39. For contrast, the annual change in PDQ-39 score across all participants averages a worsening of 1.6 points.

Thinking about what an 8 point improvement means:

In the year that a patient has a DBS device implanted, the patient has a 21% chance of experiencing an 8 point improvement in their HRQL.

The average patient has a 10.2% chance of this level of improvement.

Patients started on levodopa for the first time have a 12% chance of this level of improvement.

In the next phase of the research, we will explore: (1) Why some centers do better than others in improving HRQL with DBS (2) Can we identify the key factors in achieving the very best results with DBS (3) What are the benefits of DBS for patients who don’t get the same level of HRQL improvement (4) How do we optimize the rest of the treatment paradigm (medications, therapy, exercise, etc.)